safety & efficacy of bpl's high purity factor x in treatment of factor x deficient subjects undergoing surgery

Conditions: Factor X Deficiency

Summary: to primary efficacy variable is to assess the presence or absence of excessive blood loss during and after surgery. the secondary efficacy endpoints are as follows: 1. a subjective overall assessment by the investigator of factor x in the control of bleeding during surgery. 2. the incidence of bleeding episodes during treatment with factor x while the subject is at risk of post-operative bleeding, including location and duration. 3. incremental recovery of fx:c and fx:ag after the pre-surgery bolus infusion. 4. assessment of fx:c and fx:ag levels on each day post-surgery. 5. assessment of the cumulative weight-adjusted doses of factor x as measured by fx:c (iu/kg body weight) administered to each subject to maintain haemostasis. 6. assessment of the cumulative doses of factor x as measured by fx:c (iu) administered to each subject to maintain haemostasis. 7. amount of weight-adjusted factor x as measured by fx:c (iu/kg body weight) administered daily (day of surgery and each post-operative day) to maintain haemostasis.

to investigate the safety and efficacy of factor x administered by bolus infusion to prevent bleeding and achieve haemostasis in factor x deficient subjects undergoing surgery.

Eligibility criteria:
inclusion criteria: * subjects who are at least 12 years of age at date of written informed consent/assent. * subjects who have given written informed consent or, for subjects aged 12-17 years (inclusive), have given written assent and whose parent/guardian has given written informed consent. * subjects with hereditary mild to severe factor x deficiency (\<20% basal fx activity), including previously untreated subjects or those currently treated with fresh frozen plasma (ffp), prothrombin complex concentrate (pcc) or factor ix/x concentrate by prophylaxis or on demand. * subjects who are to undergo surgery in which the investigator believes a factor x concentrate will be required due to a prior history of unusual bleeding either spontaneously or after surgery or trauma in the absence of treatment with a factor x containing product. * pregnant subjects undergoing obstetric delivery (including caesarean surgery and vaginal delivery) may enter the study. female subjects of child-bearing potential must have a negative result on a human chorionic gonadotropin-based pregnancy test. if a female subject is or becomes sexually active, she must practice contraception by using a method of proven reliability for the duration of the study. exclusion criteria: * subjects who are required or expected to take other factor x containing medications during or after surgery. * subjects with a history of inhibitor development to fx or a detectable inhibitor to fx (≥0.6 bu) on the nijmegen-bethesda assay at screening. obtaining a fx inhibitor result at screening is not mandatory if the subject is to undergo emergency surgery and the local laboratory is unable to perform the analyses prior to the surgical procedure. * subjects with thrombocytopenia (platelets \< 50 x 109/l). * subjects who have clinically significant renal disease (creatinine \>200µmol/l). * subjects who have clinically significant liver disease (alt levels greater than three times the upper limit of normal). * subjects known to have other coagulopathy or thrombophilia. * subjects who are currently participating or have participated in another trial within the last 30 days, with the exception of the bpl factor x pk study (protocol number ten01). * female subjects who are lactating. * subjects who have known or suspected hypersensitivity to the investigational medicinal product or its excipients. * subjects known to have abused chemicals or drugs within the past 12 months. * subjects with a history of unreliability or non-cooperation.

Keywords: factor x deficiency surgery factor x deficiency subjects requiring surgery